LEXINGTON, Mass.--(EON: Enhanced Online News)--Promedior,
Inc., today announced positive data from Stage 1 of an adaptive
two-stage Phase 2 trial of PRM-151, a novel anti-fibrotic immunotherapy,
in patients with myelofibrosis. The overall response rate (ORR), defined
as IWG-MRT1 (International Working Group-Myeloproliferative
Neoplasms Research and Treatment) responses or reduction in bone marrow
fibrosis, was 43 percent at 6 months, surpassing the pre-specified
efficacy criteria necessary to proceed to the next stage of Promedior’s
Phase 2 trial. Data presented at the American Society of Hematology
(ASH) meeting demonstrate reduction of bone marrow fibrosis by at least
one grade observed in 42 percent of patients which was associated in
most patients with improvements in anemia and/or thrombocytopenia and,
in some patients, by transfusion independence lasting at least 24 weeks.
Bone marrow fibrosis grade is correlated with anemia, thrombocytopenia,
peripheral blasts and shortened survival2,3. These study
results were presented in an oral presentation by principal investigator
Srdan Verstovsek, MD, PhD, at the ASH 2014 Annual Meeting on December 8,
2014.

“We are encouraged by the favorable safety profile and clinical activity
of PRM-151 demonstrated in the first stage of this trial, and are
excited to see that a reduction of bone marrow fibrosis by PRM-151 is
associated with signs of improved hematopoiesis”

“We are encouraged by the favorable safety profile and clinical activity
of PRM-151 demonstrated in the first stage of this trial, and are
excited to see that a reduction of bone marrow fibrosis by PRM-151 is
associated with signs of improved hematopoiesis,” said Srdan Verstovsek,
MD, PhD, Professor, Department of Leukemia, Division of Cancer Medicine,
The University of Texas MD Anderson Cancer Center and Principal
Investigator for this Phase 2 trial. “There is tremendous enthusiasm for
PRM-151 among investigators and patients, and we look forward to moving
forward with Stage 2 of this important clinical trial.”

“These promising clinical data in myelofibrosis patients highlight the
differentiated benefits of PRM-151, showing an unprecedented rate of
reversing bone marrow fibrosis. Further, by validating PRM-151’s novel
mechanism of action to reverse fibrosis, this study demonstrates the
broad potential in a range of other fibrotic diseases,” said Suzanne L.
Bruhn, PhD, President and Chief Executive Officer of Promedior. “We will
continue to move PRM-151 forward as a new treatment option for patients
with myelofibrosis and other fibrotic diseases. We expect to initiate
the next stage of PRM-151’s Phase 2 clinical program in myelofibrosis in
the first half of 2015.”

In the first stage of this two-stage Phase 2 trial, 11 out of 25
evaluable patients had reduction in bone marrow fibrosis by at least one
grade, and 10 of 21 patients with baseline Hgb < 100 g/L or platelet
count < 100x109/L had substantial increases in hemoglobin
and/or platelets accompanied by transfusion independence lasting at
least 24 weeks in some patients. Improvements in symptoms, including 4
IWG-MRT Clinical Improvement Symptom responses, were also observed,
along with modest reductions in splenomegaly. Improvements were observed
in all four independent treatment groups of myelofibrosis patients who
received PRM-151 weekly or monthly and as either a single agent or in
patients showing no further improvements on a stable dose of
ruxolitinib. PRM-151 was safe and well tolerated on weekly and monthly
dosing schedules, both alone and in combination with ruxolitinib, with
no evidence of myelosuppression. Most adverse events were Grade 1 or 2
and most were considered unrelated to PRM-151. Fourteen patients are
continuing treatment in a study extension, and clinical benefits appear
to be increasing with longer treatment duration.

This Phase 2 trial is a multi-center, two stage, adaptive design study
to determine the efficacy and safety of PRM-151 as a single agent or
added to a stable dose of ruxolitinib in patients with Primary
Myelofibrosis (PMF), Post-Polycythemia Vera MF (post-PV MF), or
Post-Essential Thrombocythemia MF (post-ET MF). Twenty seven patients
were enrolled in Stage 1 of the study, additional patients will be
enrolled in Stage 2.

Myelofibrosis (MF), a type of myeloproliferative neoplasm, is a serious,
life-limiting cancer that is characterized by fibrosis of the bone
marrow. Replacement of the bone marrow by scar tissue prevents the
normal production of blood cells, leading to anemia, fatigue, and
increased risk of bleeding and infection. Data show that bone marrow
fibrosis grade is correlated with anemia, thrombocytopenia, peripheral
blasts and shortened survival2,3.

Myelofibrosis affects approximately 18,000 people per year in the U.S.,
with a median age of 61-664. The only potentially curative
treatment is allogeneic bone marrow transplant, which results in
reversal of fibrosis and normalization of blood counts, but is a
realistic option for only a small number of patients. Other currently
available therapies have minimal, if any, impact on the underlying
fibrosis, and often result in worsening in hemoglobin and platelets,
important blood parameters which are directly linked to morbidity and
mortality and remain the major unmet need in patients with MF.

In addition to the clinical study in myelofibrosis, a Phase 1b study in
patients with idiopathic pulmonary fibrosis (IPF) showed encouraging
results in exploratory efficacy endpoints, which were presented in
an oral session at the 2013 Annual Meeting of the American Thoracic
Society5.

PRM-151 has Fast Track and Orphan designation in the US for treatment of
myelofibrosis and Orphan Designation in the US and EU for treatment of
IPF.

About Promedior

Promedior
is a clinical stage immunotherapy company pioneering the development of
targeted therapeutics to treat diseases involving fibrosis. Fibrosis
occurs when healthy tissue is replaced with excessive scar tissue,
compromising function and ultimately leading to organ failure. Fibrosis
is a common feature of several rare diseases as well as more prevalent
illnesses such as age related macular degeneration, diabetic
nephropathy, nonalcoholic steatohepatitis (NASH), and several types of
solid tumors.

Promedior has advanced its lead program (PRM-151) into clinical trials
focused on two orphan fibrotic diseases, myelofibrosis and idiopathic
pulmonary fibrosis. Promedior owns world-wide rights to PRM-151 and has
a significant intellectual property estate.